2026-04-29

I know this is a topic the Minister of State is very familiar with, as she has heard me raise it many times within our parliamentary party meetings. This is something she has heard plenty of times from me in the past few years. It is something I am very passionate about. I have more or less dedicated six years of my job as a public representative to pursuing various drugs for rare diseases. I have a feeling I know what will be contained in the answer in front of the Minister of State. It will probably be something about the fact that the State is spending more money than ever on drugs for rare diseases and drugs in general. The spend is more than €4 billion. What I would like to hammer home is that a tiny fraction of that - less than 1% - is dedicated to new drugs, specifically drugs for rare diseases. When I submitted this question, I specifically wanted to talk about Friedreich's ataxia and the drug Skyclarys, which is currently under consideration by the National Centre for Pharmacoeconomics, NCPE, and the drugs group. It is a degenerative disease. I know the Minister of State is familiar with it. There are hundreds of diseases out there that would break her heart or the heart of anyone in this House if they affected a brother, sister, son or daughter of theirs. It is heartbreaking to see the stuff people are going through on a daily basis. The difficulty I have is that we have been blaming the process for too long. It clearly does not work and is fundamentally broken. At various stages, both the Fianna Fáil party leader and the Fine Gael party leader have said the process is broken. They are now in a position to change the process but we have not looked at it in a meaningful way in the six years we have been in power together. We have given a commitment in the programme for Government and I want to see us follow through on it. First, we must undertake to provide an early access scheme for people to access many of the drugs we are talking about this morning. Second, we must fundamentally overhaul the reimbursement system. They are two commitments in the programme for Government. I would like to see in the reply that the Minister has finally embarked on the task of starting that work, specifically on Skyclarys, which is currently under consideration.

The Deputy's time is up.

The NCPE is waiting for a response from the drugs company at the moment but I hope that, in the coming months, we will be in a position where the 200 patients affected will get access to the drug.

I am also here to speak about Friedreich's ataxia. On Monday evening, I met Aoife Quinn from County Galway, who was diagnosed with Friedreich’s ataxia in 2021. She outlined to me her long pathway to getting a diagnosis for this rare disease and how the disease impacted on her life. She told me of her experience dealing with symptoms like loss of balance and speech difficulties and of how her symptoms had deteriorated over time. Every day brings her a loss. It is a relentless and progressive disease. The reimbursement of Skyclarys would slow the progression of this disease and give her the chance to maintain her strength and independence for as long as possible. She is just one of approximately 200 people across the State who are living with Friedreich's ataxia. Access to this treatment would also help those who are newly diagnosed, who may be spared some of the suffering experienced by those before them. I am aware that the HSE is engaged in negotiations with the drugs manufacturer, but as of today the negotiations will have been going on for almost two years. Every single day that passes, Aoife's condition worsens, as it does for every person who is suffering from this disease. The clock is ticking for them and they cannot face more delays. This drug is an opportunity to slow the deterioration - in Aoife's case before she is wheelchair bound or suffers other issues. She does not have time to wait. We all understand that there must be a process where the HSE negotiates to get the best possible price for medicines but we need to see real urgency here because every day that passes matters. Aoife has also expressed her frustration that she is not kept updated on the negotiations and that there is not regular contact with her on that. Will the Minister of State look at that? The bottom line is that these people do not have the luxury of time. Every day that passes without the medication is a day where symptoms deteriorate. They do not have time to be left waiting in the dark, wondering whether a medicine that could improve their lives will be reimbursed. Will the Minister of State provide clarity today or give a commitment that this will be resolved soon?

I thank both Deputy O'Sullivan and Deputy O'Hara for raising this important issue. I spoke on this in the Seanad a couple of weeks ago. I acknowledge Deputy O'Sullivan's advocacy on rare diseases in the past six years. As we all know, the State acknowledges the importance of access to new and innovative medicines for patients living with a rare disease. Budgets 2021 to 2025 included dedicated funding for new medicines of €158 million. Budget 2026 allocated an additional €30 million of funding available for new drugs. From 2021 up to March 2026, this has enabled the HSE to approve reimbursement for 263 new medicines, including 72 new medicines for the treatment of rare diseases. As Members are aware, the National Rare Disease Strategy 2025-2030 was launched last year. The strategy outlines a comprehensive framework designed to enhance diagnosis, treatment and support for people living with rare diseases, aiming to improve quality of life, promote equitable access to healthcare, and foster innovation in rare disease research and treatment. It also emphasises the importance of access to orphan medicines among its key recommendations. The State has signed two new framework agreements on the supply and pricing of medicines from 2026 to 2029, following successful negotiations with the pharmaceutical industry, that will provide stability and certainty to the sector. Building on this achievement, the State and the pharmaceutical sector have agreed to establish a strategic partnership on the development of a sandbox early-access programme for rare diseases. This will be a proof-of-concept initiative aligned with the commitments in the programme for Government and the national rare disease strategy. Regarding Skyclarys, I will provide some background for our colleagues and then address the Deputy's question. Omaveloxolone, sold under the brand name Skyclarys, is a medication licensed by the European Medicines Agency to treat Friedreich’s ataxia in patients aged 16 years and over. Friedreich’s ataxia is a rare inherited disease that causes damage to the nervous system. Under the Health (Pricing and Supply of Medical Goods) Act 2013, the HSE has statutory responsibility for decisions on the pricing and reimbursement of medicines. The Act provides for a rigorous process for the assessment of new medicines for reimbursement. This ensures that the right medicines are chosen and that approval is at a sustainable price. The HSE received an application for pricing and reimbursement for Skyclarys from the applicant, Biogen Idec (Ireland) Limited, on 1 August 2024. After receiving a completed rapid review assessment report from the National Centre for Pharmacoeconomics, the HSE commissioned a full health technology assessment on 25 September 2024. A fully completed dossier, which is required to allow the NCPE to conduct a pharmacoeconomic assessment of the pricing and reimbursement application, was not received from Biogen until July 2025. The NCPE proactively engaged with the applicant company and submitted the finalised health technology assessment report to the HSE on 16 December 2025. It is important that we look at the timelines. This report included submissions made by patient organisations. These submissions will form part of the data that the HSE considers. The HSE invited the applicant to commence price negotiations and offered a range of dates to the company. The HSE has advised this meeting took place on 12 February 2026 and, as of the most recent update from the HSE in April, a commercial proposal from the company remains outstanding. I checked with the Minister's Department last night in this regard and I know the Taoiseach has engaged with her. Where we are at the moment is that a commercial proposal from the company remains outstanding, which I know is frustrating, but it is important to put the timelines on the record of the Dáil.

I thank the Minister of State for the response. It is predictable, although I do not say that to be dismissive. I know a lot of this and have known it for some time. The point I made in my initial salvo was that we rank 23rd out of 27 EU countries with regard to reimbursement for rare disease drugs. That is a fact. Whether it is Skyclarys or some other drug for some other ailment, the fact of the matter is that Ireland is a laggard among our EU counterparts in terms of reimbursement. I understand that engagement with this company has been attempted. I have not come across this company in the past. I wrote to it myself last week to look for engagement. I would like to think it might do something immediate, short-term and local with patients to give them early access in the absence of State intervention. However, fundamentally, we must be truthful with ourselves in here and recognise that the system is flawed. It is the same system we have had for decades. Any common drug or other product on the shelf of a pharmacy that costs you €2 or €3 goes through the same process as one of these drugs that costs hundreds of thousands of euro. If we fail to acknowledge that fundamental flaw in the system, this issue is going to keep being repeated.

I appreciate the update from the Minister of State. I cannot emphasise enough the impact the ongoing delays are having on those impacted by the disease. I also call on the drug company to engage in good faith in the negotiations with the HSE. The priority of all parties involved should be delivering this vital medication to the people who need it. Aoife and all of those who suffer from Friedreich’s ataxia want an opportunity to have as good a quality of life as possible. This medication is critical for them. As we speak, people like Aoife are losing their mobility, their ability to live independently and so on. The onus is on the Minister and the Department to ensure that people who suffer from rare diseases are given access to vital medication. The frustration is that we are one of the wealthiest countries in the world and this drug is available in many other European countries. The Minister of State mentioned that the commercial proposal is still outstanding. It has been two years since this process commenced. If she were to put herself in the shoes of somebody like Aoife, she would see how there would be enormous frustration at the delays that are happening here. I urge her to do absolutely everything she can to get this resolved as soon as possible. Every single day matters.

I concur with both Deputies. We are speaking about this really important drug and 200 people, their families and their parents want to know what is going to happen and are hanging on our every word. I take all that on board. While I know this has been going on for two years, the HSE did not receive the information from Biogen until July 2025. We are still waiting for the commercial proposal from the company. That remains outstanding. That is the situation at the moment. I am glad Deputy O'Sullivan has engaged with the company to see if we could get some movement on this. We have an agreement in relation to some drugs. We have the Benelux agreement, which allows us to get really good value in procuring drugs. However, it is different for drugs for rare diseases. You need a number of elements to work together. I agree with Deputy O'Sullivan about the overall system, the process and meaningful engagement. I will be speaking to the Minister about this again. The sooner the commercial proposal from the company comes in, the sooner a decision can be made. There is not a Deputy in the House who has not been touched by Aoife's case and those of others who are awaiting this drug. Did the Deputies get my response? Was it circulated?

I have the response from the Minister of State.